A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00943111
First received: July 20, 2009
Last updated: March 7, 2014
Last verified: March 2014
  Purpose

This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in patients with Gaucher disease type 1 who have reached therapeutic goals with enzyme replacement therapy.


Condition Intervention Phase
Gaucher Disease, Type 1
Drug: eliglustat tartrate
Drug: imiglucerase for injection
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Multi-Center, Multi-National, Open-Label, Active Comparator Study to Evaluate the Efficacy and Safety of Genz-112638 in Patients With Gaucher Disease Type 1 Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy

Resource links provided by NLM:


Further study details as provided by Sanofi:

Primary Outcome Measures:
  • The percentage of patients who remain stable for 52 weeks (the primary analysis period) [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Absolute change in total T- and Z-scores for bone mineral density from baseline to week 52 [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Absolute change in hemoglobin levels (g/dL) from baseline to week 52 [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Percent change in platelet counts from baseline to week 52 [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Percent change in spleen volume (in multiples of normal (MN)) from baseline to week 52 [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Percent change in liver volume (in MN) from baseline to week 52 [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]

Enrollment: 160
Study Start Date: October 2009
Estimated Study Completion Date: May 2015
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Investigational
eliglustat tartrate (Genz-112638)
Drug: eliglustat tartrate
Capsules: 50, 100, or 150 mg BID (twice daily)
Other Name: Genz-112638
Active Comparator: imiglucerase for injection Drug: imiglucerase for injection
Intravenous (IV) Infusion, Varied Dose, Q2(bi-weekly)
Other Name: Cerezyme

Detailed Description:

Gaucher disease is characterized by lysosomal accumulation of glucosylceramide due to impaired glucosylceramide hydrolysis. Gaucher disease type 1, which is the most common form, accounts for >90% of cases and does not involve the CNS. Typical manifestations of Gaucher disease type 1 include splenomegaly, hepatomegaly, thrombocytopenia, anemia, bone disease, and decreased quality of life. The disease manifestations are caused by the accumulation of glucosylceramide (storage material) in macrophages (called Gaucher cells) which have infiltrated the spleen and liver as well as other tissues.

Eliglustat tartrate (Genz-112638) is a small molecule drug developed as an oral therapy which acts to specifically inhibit production of this storage material in Gaucher cells.

This study is designed to determine the efficacy, safety, and pharmacokinetics (PK) of eliglustat tartrate (Genz-112638) in adult patients with Gaucher disease type 1 who have been stabilized on enzyme replacement therapy.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • The patient (and/or their parent/legal guardian) is willing and able to provide signed informed consent prior to any study-related procedures to be performed.
  • The patient is at least 18 years old at the time of randomization.
  • The patient has a confirmed diagnosis of Gaucher disease type 1.
  • The patient has received treatment with enzyme replacement therapy (ERT) for at least 3 years. Within the 9 months prior to randomization, the patient has received a total monthly dose of 30 to 130 U/kg for at least 6 months.
  • The patient has reached Gaucher disease therapeutic goals prior to randomization.
  • Female patients of childbearing potential must have a documented negative pregnancy test prior to dosing. In addition, all female patients of childbearing potential must use a medically accepted form of contraception throughout the study.

Exclusion Criteria:

  • The patient has had a partial or total splenectomy within 3 years prior to randomization.
  • The patient has received substrate reduction therapies for Gaucher disease within 6 months prior to randomization.
  • The patient has Gaucher disease type 2 or 3 or is suspected of having Gaucher disease type 3.
  • The patient has any clinically significant disease, other than Gaucher disease, including cardiovascular, renal, hepatic, gastrointestinal (GI), pulmonary, neurologic, endocrine, metabolic (e.g. hypokalemia, hypomagnesemia), or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may confound the study results or, in the opinion of the Investigator, may preclude participation in the study.
  • The patient has tested positive for the human immunodeficiency virus (HIV) antibody, Hepatitis C antibody, or Hepatitis B surface antigen.
  • The patient has received an investigational product within 30 days prior to randomization.
  • The patient is pregnant or lactating.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00943111

  Show 34 Study Locations
Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Additional Information:
Publications:
Responsible Party: Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier: NCT00943111     History of Changes
Other Study ID Numbers: GZGD02607, 2008-005223-28
Study First Received: July 20, 2009
Last Updated: March 7, 2014
Health Authority: United States: Food and Drug Administration
Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
Australia: Therapeutic Goods Administration'Brazil: Ministry of Health
Brazil: Ministry of Health
Canada: Health Canada
Egypt: Ministry of Health and Population
France: Ministry of Health
Germany: Ministry of Health
Italy: Ministry of Health
Russia: Ministry of Health of the Russian Federation
Spain: Ministry of Health
United Kingdom: Medicines and Healthcare Products Regulatory Agency

Keywords provided by Sanofi:
Gaucher disease,
Genz-112638,
beta-glucosidase,
acid ß-glucosidase,
glucocerebrosidase,
glucosylceramide,
D-glucosyl-N-acylsphingosine glucohydrolase,
substrate reduction therapy

Additional relevant MeSH terms:
Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on July 29, 2014